A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
- Conditions
- Cystic Fibrosis
- Interventions
- Genetic: BI 3720931Drug: PlaceboDevice: Inhaler
- Registration Number
- NCT06515002
- Lead Sponsor
- Boehringer Ingelheim
- Brief Summary
This study is open to adult men with cystic fibrosis and adult women with cystic fibrosis who cannot have children. People with cystic fibrosis can join if they are not eligible to receive cystic fibrosis transmembrane conductance regulator modulator therapy (CFTR-MT). The purpose of this study is to find out how well a medicine called BI 3720931 is tolerated and whether it improves lung function in people with cystic fibrosis. In this study, BI 3720931 is given to humans for the first time.
This study has two phases. In Phase 1, participants are put in one of 3 groups, one group after the other. Each group gets a different dose of BI 3720931. Group 1 starts with the lowest dose, followed by group 2 with the middle and group 3 with the high dose. In Phase 2, participants are put into 3 groups by chance, but at the same time. 2 groups get different doses of BI 3720931, and 1 group gets placebo. All study participants get only 1 dose of BI 3720931 or placebo and they use a special inhaler to take the study medicine. The placebo inhaler looks like the BI 3720931 inhaler but does not contain any medicine. During the study, participants continue taking their usual medicines.
Doctors closely monitor participants' health at the study site for the first 3 days after receiving BI 3720931. Participants visit their doctors regularly thereafter. The doctors check the health of the participants and note any health problems that could have been caused by BI 3720931. Study participants regularly do a standard lung function test to measure how well their lungs are working. Participants, in either Phase 1 or Phase 2, are in the study for 7 months. After completion of this study, participants will take part in a long-term follow-up study (1504-0003).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 36
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Phase I: Dose group 1 (low dose) BI 3720931 - Phase II: Dose group 1 BI 3720931 - Phase II: Dose group 1 Inhaler - Phase II: Placebo group Inhaler - Phase I: Dose group 1 (low dose) Inhaler - Phase I: Dose group 3 (high dose) BI 3720931 - Phase I: Dose group 2 (medium dose) BI 3720931 - Phase II: Dose group 2 Inhaler - Phase I: Dose group 3 (high dose) Inhaler - Phase II: Dose group 2 BI 3720931 - Phase I: Dose group 2 (medium dose) Inhaler - Phase II: Placebo group Placebo -
- Primary Outcome Measures
Name Time Method Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration up to 24 weeks Phase II: Absolute change from baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp) at Week 8 after drug administration at baseline, at week 8
- Secondary Outcome Measures
Name Time Method Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration at baseline, at week 24 Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration up to 24 weeks Phase I: Occurrence of treatment response defined as change from baseline ≥5% in FEV1pp, comparing the mean of 3 pre-treatment FEV1pp measured in the screening period with the mean of 3 post-treatment FEV1pp-values at Weeks 4, 6, and 8 up to 12 weeks Phase I: Occurrence of any dose limiting toxicities (DLTs) up to Week 24 after drug administration up to 24 weeks Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration at baseline, at week 24 Phase II: Occurrence of any serious adverse events (SAEs) up to Week 24 after drug administration up to 24 weeks
Trial Locations
- Locations (10)
HOP Gui de Chauliac
🇫🇷Montpellier, France
University Hospital Llandough
🇬🇧Cardiff, United Kingdom
Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
HOP Necker
🇫🇷Paris, France
Osp. Pediatrico Bambin Gesù
🇮🇹Roma, Italy
Istituto G. Gaslini
🇮🇹Genova, Italy
Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
Royal Brompton Hospital
🇬🇧London, United Kingdom
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom